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Journal article
Finding the optimal regimen for Mycobacteroides abscessus treatment (FORMaT) in people with Mycobacteroides abscessus pulmonary disease: a multicentre, randomised, multi-arm, adaptive platform trial
BMJ Open, Vol.15(9), pp.1-16
2025
PMID: 40976660
Abstract
Introduction: Mycobacteroides abscessus (MABS) is within the non-tuberculous mycobacteria family. It inhabits soil and water, exhibits multi-antibiotic resistance and causes opportunistic lung infections, which may progress to symptomatic MABS-pulmonary disease (MABS-PD) associated with substantial morbidity, increased healthcare utilisation, impaired quality of life and increased mortality. Treatment regimens for MABS-PD are highly variable, not evidence-based and involve complex, expensive drug combinations administered for prolonged periods (>12 months) with frequent adverse effects and treatment failure. There is an urgent need for safe, efficacious and cost-effective MABS-PD therapy. Here, we describe the Master Protocol for the Finding the Optimal Regimen for Mycobacteroides abscessus Treatment (FORMaT) trial. FORMaT aims to determine the most effective and best tolerated treatment for MABS-PD as defined by MABS clearance from respiratory samples with good treatment tolerance.
Methods and analysis: FORMaT is an international multicentre, adaptive platform trial evaluating treatment combinations for MABS-PD. Participants are randomised multiple times during the trial, with assessment of the primary outcome of clearance of MABS infection with good treatment tolerance. Initially, therapies recommended in international consensus guidelines are being tested. Data obtained will eliminate therapies lacking efficacy or causing unacceptable toxicity. Novel treatments can then be added and tested against previously determined optimal approaches, leading in an iterative fashion to improved microbiological clearance and health outcomes. In parallel, an Observational cohort and several integrated and discovery studies are embedded in FORMaT to identify biomarkers of MABS-PD and MABS clearance, clinical and radiographic treatment response, drug pharmacokinetics and Mycobacteroides genomics and resistome.
Ethics and dissemination: The FORMaT Master Protocol and related documents are approved by regulatory authorities in each participating jurisdiction and/or site. Results will be published in peer-reviewed journals and presented at scientific meetings. De-identified, aggregated data will be shared on an approved online platform.T
Details
- Title
- Finding the optimal regimen for Mycobacteroides abscessus treatment (FORMaT) in people with Mycobacteroides abscessus pulmonary disease: a multicentre, randomised, multi-arm, adaptive platform trial
- Authors
- Tiffany Jong (Corresponding Author) - The University of QueenslandTimothy Baird - University of the Sunshine Coast, Queensland, Centre for BioinnovationHelen Louise Barr - Nottingham Biomedical Research CentreScott Bell (Author) - The University of QueenslandTheophile Bigirumurame - Newcastle UniversityKara Brady - The University of QueenslandAndrew Burke (Author) - The University of QueenslandJoshua Byrnes - Griffith UniversityDaan Caudri - Erasmus MCJulia E. Clark (Author) - The University of QueenslandLachlan J. M. Coin (Author) - The University of MelbourneFelicia Goh - The University of QueenslandKeith Grimwood - Griffith UniversityDaniel Hicks - The University of QueenslandKaushala Jayawardana - Murdoch Children's Research InstituteSri Joshi - Murdoch Children's Research InstituteKatherine Lee (Author) - The University of MelbourneJason A. Roberts (Author) - The University of QueenslandTavs Qvist - RigshospitaletGeraint Rogers - Flinders UniversityDavid Reid (Author) - Prince Charles HospitalClaire Shackleton - The Kids Research Institute AustraliaMegan Rice - The University of QueenslandPeter D. Sly (Author) - Children's Health Queensland Hospital and Health ServiceAlan R. Smyth - Queen's University BelfastLuke Stevens - Murdoch Children's Research InstituteRebecca Stockwell - The University of QueenslandAbdullah Tarique - The University of QueenslandSteven Taylor - South Australian Health and Medical Research InstituteRachel Thomson (Author) - The University of QueenslandHarm A. W. M. Tiddens - Erasmus MCXiao Fang Wang - Murdoch Children's Research InstituteJames Wason (Author) - Newcastle UniversityClaire Wainwright - The University of Queensland
- Publication details
- BMJ Open, Vol.15(9), pp.1-16
- Publisher
- BMJ Group
- Date published
- 2025
- DOI
- 10.1136/bmjopen-2024-096188
- ISSN
- 2044-6055
- PMID
- 40976660
- Copyright note
- © Author(s) (or their employer(s)) 2025. Reuse permitted under CC BY- NC. No commercial re-use. See rights and permissions. Published by BMJ Group.
- Grant note
- FORMaT is funded by the Australian Government Department of Health and Aged Care through the Medical Research Future Fund (MRFF) (2017/ MRF1152249); the US Cystic Fibrosis Foundation (WAINWR19A0); the Children’s Hospital Foundation (50301); the University of Queensland; an anonymous donor and the Thoracic Society of Australia and New Zealand.
- Organisation Unit
- School of Science, Technology and Engineering; Centre for Bioinnovation
- Language
- English
- Record Identifier
- 991172845402621
- Output Type
- Journal article
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